In fewer than five years, Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR genome-editing technology, has taken the scientific community by storm, not only revolutionizing the pace of biological breakthroughs but also promising medicines that cure diseases now considered incurable. In the coming weeks, ARK will publish a white paper based on original research exploring the significance of CRISPR and the investment opportunities it will create. Until then, please enjoy a primer to assist in understanding DNA, the human genome, and the rise of CRISPR.
The structure of DNA, the double-stranded helix
The human genome is composed of 3.2 billion base pairs of DNA, or lines of code, a single error in which could cause disease.1,2 Biological tools are evolving that should be able to correct these errors, or mutations, easily and cost-effectively, making cures for diseases possible for the first time in history.
The ability to control genes holds the promise of a cure for any disease. The 23,000 genes in each human genome contain instructions that guide the production of proteins, which are the building blocks that govern the functioning of the human body. Proteins fight infections, carry oxygen, clot blood after injury, and control muscle movements. They regulate brain signals, trigger smells, and form finger and toe nails. Each of these functions depends upon precise amounts of specific proteins.
The Rise of CRISPR
Gene-editing debuted during the biotechnology revolution. As shown below, the first targeted edit was in a yeast cell during the 1980s. Major therapeutic breakthroughs associated with gene-editing have taken place only in the past decade. Today, genome-editing is a rapidly developing field for 21stcentury medicine.
CRISPR is a simple, powerful and programmable genome-editing tool. An acronym for Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR was discovered in a study of viral invaders and bacterial immune defense systems.3
According to ARK’s research, unlike previous gene splicing tools, not only does CRISPR have a wide range of applications, but it also is accessible and fast. While associated primarily with gene-editing today, CRISPR ultimately will be able to control gene processes and protein expression. As a result, CRISPR is evolving into a novel technology platform in the bioengineering space, accelerating innovation in a number of areas, including:
- Basic Research
- Drug Discovery/ Animal Models
- Drug Development/ Therapeutics
- Data Storage
- Material Sciences
For an in depth review of CRISPR and the investment opportunities it creates, subscribe below to be notified of the publication of ARK’s Research Whitepaper.
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